SAN RAFAEL, Calif., Feb. 19, 2013 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced today results from the PKU-016 ASCEND study, the largest randomized controlled trial evaluating neuropsychiatric outcomes in phenylketonuria (PKU) patients treated with the approved drug Kuvan (sapropterin dihydrochloride).
The study evaluated medically important symptoms similar to attention deficit hyperactivity disorder (ADHD) in PKU patients whose blood levels of phenylalanine (Phe) are reduced by Kuvan. The primary endpoint of the study was evaluated using an attention deficit hyperactivity rating scale (ADHD-RS), commonly used to evaluate symptoms of inattentiveness and hyperactivity. Kuvan improved the ADHD-RS (p=0.085), driven by a statistically significant change in the inattention component of the score (p=0.036). PKU patients have a lower degree of hyperactivity as compared to ADHD patients, which likely resulted in the less prominent benefits in this domain. The neurocognitive benefit of Kuvan in patients who respond to Kuvan was corroborated by the Behavior Rating Inventory of Executive Function, or BRIEF, in children who were rated by their parents. Moderate to severe inattention deficit affected approximately one-third of patients in the study. Preliminary analysis of safety indicates that it is consistent with the general experience with Kuvan.
"We are encouraged to see the positive trend in the predetermined ADHD primary endpoint, which was driven by a statistically significant change in the sub-domain of inattentiveness," said Hank Fuchs, M.D., Chief Medical Officer of BioMarin. "We believe these results will provide useful information to the medical community on clinically relevant benefits of Kuvan and facilitate adoption of the product. Further, the results will help inform the design of the Phase 3 PEG-PAL program with key learnings about the measurement tools. We are encouraged by the results and plan to discuss the submission of this data with the FDA for possible inclusion in the Kuvan label."In addition to the ADHD-RS and BRIEF instruments, other exploratory tests in the PKU-016 study of neuropsychiatric deficit in PKU patients demonstrated a higher level of impairment in PKU patients than the normal population. Further analyses are underway to complete the evaluation of Kuvan on these outcome measures.
Select the service that is right for you!COMPARE ALL SERVICES
Jim Cramer and Stephanie Link actively manage a real portfolio and reveal their money management tactics while giving advanced notice before every trade.
- $2.5+ million portfolio
- Large-cap and dividend focus
- Intraday trade alerts from Cramer
- Weekly roundups
Access the tool that DOMINATES the Russell 2000 and the S&P 500.
- Buy, hold, or sell recommendations for over 4,300 stocks
- Unlimited research reports on your favorite stocks
- A custom stock screener
- Upgrade/downgrade alerts
Jim Cramer's protege, David Peltier, identifies the best of breed dividend stocks that will pay a reliable AND significant income stream.
- Diversified model portfolio of dividend stocks
- Alerts when market news affect the portfolio
- Bi-weekly updates with exact steps to take - BUY, HOLD, SELL
All of Real Money, plus 15 more of Wall Street's sharpest minds delivering actionable trading ideas, a comprehensive look at the market, and fundamental and technical analysis.
- Real Money + Doug Kass Plus 15 more Wall Street Pros
- Intraday commentary & news
- Ultra-actionable trading ideas
Our options trading pros provide daily market commentary and over 100 monthly option trading ideas and strategies to help you become a well-seasoned trader.
- 100+ monthly options trading ideas
- Actionable options commentary & news
- Real-time trading community
- Options TV